4:49 pm

Sat February 2, 2013

FDA Challenges Stem Cell Companies As Patients Run Out Of Time

Scientists have seen promise in the potential of stem cells, but not everyone agrees stem cell replacement therapy is ready for prime time.

U.S. Food and Drug Administration

Americans seeking stem cell replacement therapy hope the process can heal them of myriad diseases, and a 2011 report by the Baker Institute estimated the industry could bring in $16 billion in revenue by 2020.

But the Food and Drug Administration has expressed concerns. The agency sued to stop treatments at a Regenerative Sciences clinic in Colorado and closed the lab of another company, Celltex Therapeutics, in Texas.

While patients in dire situations are willing to try the therapy (and pay the hefty costs), there's concern the research doesn't support broad practice just yet.

Running Out Of Options

SammyJo Wilkinson remembers the day 18 years ago when she suddenly couldn't stand. She was a young, ambitious computer programmer, launching an Internet startup.

Her doctor told her it was secondary progressive multiple sclerosis.

Now, at 48, Wilkinson is in a wheelchair. She's tried every available FDA-approved drug and two experimental therapies, including a two-year course of chemotherapy. Nothing worked.

Over time, she started researching adult stem cell replacement, but it was available only abroad. The U.S. opened its doors to the treatment two years ago.

Wilkinson had to see a doctor first, but then she paid Celltex $28,000 for stem cell replacement. In a nutshell, the company promises to remove good, healthy stem cells; store them; make more; and put them back into her body where they can help her heal.

"What the FDA said is what Celltex does to the stem cells is more than 'minimally manipulated,' which means that when they put the stem cells in that medium, they're doing something to it, and that process needs to be regulated," she tells Sullivan.

She says that what Celltex argues is that what it offers should be considered a medical procedure, rather than a product or drug (which would have to be approved and regulated by the FDA).

"But the FDA wasn't satisfied that what Celltex was promising the patients is what the patients were getting," Berfield says.

Andrea Ferrenz, executive vice president and legal counsel for Celltex, says the company is not selling a cure to sick people. She says the company provides treatment only when doctors request the company's services for their patients.

One of the doctors who refers patients is Celltex co-founder Stanley Jones. Ferrenz says this is not a conflict and that Jones is one of many doctors who want their patients to undergo adult stem cell replacement. He is also a patient himself for his arthritis.

The company is keeping a research arm open in the United States, but the profit-making part of the company is headed to Mexico, Ferrenz says, where it's opening a new clinic to expand the growing business.

"COFEPRIS, Mexico's equivalent of the FDA, is as concerned with safety for the people in its country as the U.S. FDA," she says.

Analyzing The Data

Ferrenz cites studies the company has done and some independent research that suggests the treatments are effective. But many scientists disagree. Among the skeptics is Dr. George Daley, who works at Boston Children's Hospital and is a founder of the Harvard Stem Cell Institute.

"There's a lot of promise, but we are still in the earliest experimental stages. The data, even in animals, has been highly variable," he says. "Certainly, we don't have convincing, compelling evidence that these cells work in humans, certainly not for the vast array of diseases that seem to be being treated at this time."

Daley says the therapy does have a lot of promise, though. It's even used at Children's Hospital, but only with certain cancers and diseases doctors are sure it treats.

Daley says stem cell therapy could become an everyday practice in a few decades.

"We need decades to learn how to harness the healing power of stem cells," he says.

No Time Left

For MS patients like Wilkinson, the future may be too far away.

"I don't have time," she says. "I'm completely out of time."

Wilkinson recognizes that the FDA has to do its job and that Celltex has to protect itself as well. But, she says, in the end, there will only be one loser in this fight over stem cells.

"[My husband and I have] decided ... through endless hours of discussion that this is the best investment we can make to improve both our lives," she says. "We're always just waiting in hope."

That's exactly what critics of these companies are worried about.

Copyright 2013 NPR. To see more, visit http://www.npr.org/.

Transcript

LAURA SULLIVAN, HOST:

It's WEEKENDS on ALL THINGS CONSIDERED from NPR News. I'm Laura Sullivan.

SULLIVAN: That's no preacher selling snake oil. That's Texas Governor Rick Perry counting the benefits of a controversial therapy called adult stem cell replacement. In 2011, Perry underwent back surgery, and he says the treatment aided his recovery. He's been a convert ever since, and he's not the only one. Many Americans are hoping this process can heal them of a host of diseases.

And it's estimated the industry could be a $16 billion business by 2020. But the FDA has expressed concerns. They've shut down one company in Colorado already, and they've closed the lab of another company in Texas. And that's our cover story today.

Eighteen years ago, SammyJo Wilkinson was a young ambitious computer programmer launching an Internet start-up when suddenly she collapsed. Her doctor told her it was secondary progressive multiple sclerosis.

SAMMYJO WILKINSON: I know it's just devastating. It's a very scary diagnosis because you know that the long-term outlook is that you might end up, you know, in a wheelchair.

SULLIVAN: Now at 48, Wilkinson is in a wheelchair. She has tried every available FDA-approved drug and two experimental therapies, including a two-year course of chemotherapy.

WILKINSON: You know, it left tracks on my arms like a junky and decreased my heart function. I had to actually go off it a year early because of the heart damage.

SULLIVAN: Here's what you need to know about SammyJo Wilkinson: She's read every word of published literature on MS. She even talks in the language of medical researchers. She wanted to try stem cells, but she didn't want to go abroad, so she waited, she and her husband Doug, biding their time saving their money.

And then two years ago, a U.S. company opened its doors, a Houston company called Celltex. She called them up. They told her she would need a doctor's approval. And then they gave her a recommendation of who she should see.

WILKINSON: I flew to Houston. I wouldn't know where to go find a doctor from out of town.

SULLIVAN: So who was the doctor that prescribed the treatment for you?

WILKINSON: Dr. Jones.

SULLIVAN: As I'm sure you know, Dr. Stanley Jones is one of the founders of Celltex.

WILKINSON: Yes. And he's also a personal investor in this.

SULLIVAN: Did that raise any red flags for you?

WILKINSON: You know, the commercial side of this is just a fact of life. And thank God for it because, you know, if there's a financial incentive that is going to encourage doctors to bring a lifesaving therapy to this country and make it available, then I'm all for it.

SULLIVAN: Wilkinson paid $28,000 for her stem cell treatment. In a nutshell, the company promises to remove good healthy stem cells, store them, make more and put them back into her body. Wilkinson says she believes that she got better immediately. She says 11 of her 25 MS-related symptoms have either improved or disappeared completely. She was all set to go back and do it again, and then the FDA shut down the Celltex lab.

Susan Berfield is a writer for Bloomberg Businessweek. And she wrote an article for the magazine last month, investigating the FDA's case against Celltex.

SUSAN BERNFIELD: What the FDA said is what Celltex does to the stem cells, it's more than minimally manipulated, which means when they put the stem cells in that medium, they're doing something to it, and that process needs to be regulated. What Celltex argues is that we're not doing anything more than mixing it with some stuff and putting it right back in the patients, and that that should just be considered a medical procedure, not a product, not a drug. But the FDA wasn't satisfied that what Celltex was promising patients is what the patients were getting.

SULLIVAN: Is there any way for them to know exactly what they're getting? I mean, is there a chance that without their knowledge, they could possibly be getting a bag of sugar?

BERNFIELD: There is a chance, and the FDA's report on inspection of Celltex's lab raised those kinds of questions. Celltex has since opened up discussions with the FDA, but at the same time they said that they will begin to comply with FDA regulations and that they will begin an early-stage clinical trial. So they've acknowledged the FDA's, you know, sovereignty, if you will, over them and what they are doing in Texas.

SULLIVAN: So what are they going to do in the meantime?

BERNFIELD: This week, they sent an email to their patients and let them know that Celltex was going to start working with doctors in Mexico. They will also open another lab in Houston where the company is based. And though they weren't clear about it, it seems that they will use that lab to conduct the FDA trials and research. And at the same time they'll be offering treatments in Mexico.

SULLIVAN: We reached out to Celltex to see if we could talk to co-founder Dr. Stanley Jones or someone else at the company about the work that they do. They put us in touch with their lawyer, Andrea Ferrenz. She represents Celltex before the FDA. Ferrenz says Celltex is not selling a cure to sick people. She says the company only provides treatment when independent doctors request the company's services for their patients.

FERRENZ: He is an independent physician. He does not have a clinic here at Celltex. He is on our board of directors, but he does not operate out of Celltex. He has his own spinal surgery practice.

SULLIVAN: Does Dr. Jones send his patients to Celltex?

FERRENZ: He has had patients that come to Celltex that he ordered products for, but we have other physicians as well that order products through Celltex.

SULLIVAN: Ferrenz says this is not a conflict. Dr. Jones is only one of many doctors who want their patients to undergo adult stem cell replacement. And she says Dr. Jones is a patient himself. He has said that stem cell replacement cured him of arthritis. Ferrenz says the move to Mexico is about expanding their growing business.

FERRENZ: We're bringing this technology to Mexico. It would be through physicians in Mexico.

SULLIVAN: You know, a lot of people might hear that and think of, you know, a guy behind a door on a street in Mexico, you know, who says he's a doctor. And if you want to get the Celltex process, you just knock on his door and he'll send you over to Celltex. I mean, there's something that sounds like there's less regulation perhaps in that process than there might have been in the United States.

FERRENZ: It's unfortunate that that might be the impression people have. We have met with very highly credible physicians and will be working with very highly credible physicians and excellent hospitals that are in Mexico. And COFEPRIS, Mexico's equivalent of the FDA, is as concerned with safety for the people in its country as the USFDA.

SULLIVAN: Celltex's Andrea Ferrenz cites studies the company has done and some independent research that suggests the treatments are effective. But Dr. George Daley, one of the founders of the Harvard Stem Cell Institute, disagrees.

DR. GEORGE DALEY: There's a lot of promise, but we are still in the earliest experimental stages. Certainly, we don't have convincing, compelling evidence that these cells work in humans, certainly not for the vast array of diseases that seem to be being treated at this time.

SULLIVAN: So what's your take on these companies that are selling this treatment as a way to cure disease?

DALEY: Oh, I'm deeply concerned, deeply, deeply concerned because I think they are getting way ahead of what we understand. We are not even yet completely comfortable that these cells can be delivered safely in many, many different circumstances. We know that cell preparations like the ones that are being infused have resulted in things like clots that have led to patients dying.

When they're injected in spaces of the body where they don't typically exist, like the central nervous system, we know that they can cause all kinds of damage. We're starting to see reports in the medical literature of some of the complications. So I think that the patients who are otherwise desperate, you know, they're willing to bear any risk, pay any price, they have to realize that these are experimental. And, in fact, the treatments could make their disease or their condition worse.

SULLIVAN: What do you say to people who said, look, you know, I think that this is working for me? You know, it's my money. I don't think that this is hurting me. I just really think the FDA needs to get out of the way.

DALEY: You know, I wish that we could make medical advances by anecdote alone. We can't. The fact is that when people have a financial stake in the outcomes of clinical trials, it's very hard to see them objectively assessing the outcomes. When patients are driven and feel so inclined to pay tens of thousands of dollars for a treatment, they're bound to think that they're going to get some benefit, which something we've appreciated in medicine for many, many years - a placebo effect.

SULLIVAN: Do you think that it's going to be a treatment that we're going to see used normally on lots of patients in the future?

DALEY: Well, I'm hugely optimistic. I mean, I direct our Stem Cell Transplantation Program at the Children's Hospital. We use primarily hematopoietic or blood stem cells. We do it from bone marrow. We do it from core blood, and we use it to treat leukemias and some solid cancers like neuroblastoma and a variety of genetic diseases like immune deficiency and sickle-cell anemia. We know it works there. And it's really the only established accepted standard of care using adult stem cells. Everything else is the realm of experimentation today.

I believe, truly, if we look into the future, maybe 20, 30, 40 years, cells are going to be delivered as medicines, and it will be part of our everyday practice. But it takes decades.

SULLIVAN: For SammyJo Wilkinson, that may be a bit too long.

WILKINSON: I don't have time. I'm completely out of time.

SULLIVAN: Wilkinson says she knows the FDA has to do its job, and she knows Celltex is in the business to make money. But she just wants to live.

WILKINSON: We've decided, you know, through endless hours of discussion that this is the best investment we can make to improve both our lives, you know, because it's no fun being a caretaker of a disabled wife. I mean, he has to do all the housework, all the cleaning, all the cooking, all the shopping. And I can't do anything. So it just, you know, creates an unfortunate unbalanced lifestyle. And we're always just waiting in hope.

SULLIVAN: And that's exactly what critics of these companies are worried about.

(SOUNDBITE OF MUSIC)

SULLIVAN: You're listening to ALL THINGS CONSIDERED from NPR News. Transcript provided by NPR, Copyright NPR.